The safety and effectiveness of genetically corrected iPSCs derived from β-thalassaemia patients in nonmyeloablative β-thalassaemic mice

BACKGROUND: β-Thalassaemia is a clinically common cause of hereditary haemolytic anaemia stemming from mutations in important functional regions of the β-globin gene. The rapid development of gene editing technology and induced pluripotent stem cell (iPSC)-derived haematopoietic stem cell (HSC) tran...

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Publicado en:Stem Cell Res Ther
Autores principales: Xian, Yexing, Xie, Yingjun, Song, Bing, Ou, Zhanhui, Ouyang, Shuming, Xie, Yuhuan, Yang, Yi, Xiong, Zeyu, Li, Haoxian, Sun, Xiaofang
Formato: Artigo
Lenguaje:Inglês
Publicado: BioMed Central 2020
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Research
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7367314/
https://ncbi.nlm.nih.gov/pubmed/32678022
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13287-020-01765-w
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